April 9, 2021April 9, 2021
‘Break a Sweat for Fabry’ and Other Activities Mark Awareness Month
News
Activities ranging from sharing educational posters to “breaking a sweat” are underway to mark Fabry Disease Awareness Month, set aside ... Read more
April 2, 2021April 2, 2021
Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds
News
As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an ... Read more
March 26, 2021March 26, 2021
Fabrazyme’s Long-term Benefits to Kidneys, Other Organs Added to US Label
News
Long-term treatment with Fabrazyme (agalsidase beta), an enzyme replacement therapy for people with Fabry disease, slows the progression of kidney disease and ... Read more
March 24, 2021March 23, 2021
Regaining My Strength and Stamina Following Heart Transplant
Columns, Fabry: A Road Less Traveled – a Column by Jerry Walter
I never imagined my first episode of irregular heartbeats, or arrhythmias, in the late 1970s would lead to getting a ... Read more
March 19, 2021March 19, 2021
NanoGLA, Possibly More Effective ERT for Fabry, Named Orphan Drug in EU
News
NanoGLA — a potentially more effective version of enzyme replacement therapy that delivers the missing enzyme in Fabry disease through tiny ... Read more
March 12, 2021
How Family Members Can Help Fabry Disease Patients
Health Insights
Fabry disease is a progressive, rare, genetic disorder that can lead to a number of health issues that affect a ... Read more
March 12, 2021March 12, 2021
PRX-102 Safe and Effective Once Monthly Fabry Treatment, Phase 3 Data Show
News
A once-a-month regimen of the investigational enzyme replacement therapy (ERT) PRX-102 (pegunigalsidase alfa) is safe and effective at maintaining disease ... Read more
March 8, 2021March 8, 2021
Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day
News, syndicated
Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, ... Read more
March 5, 2021March 5, 2021
FAQs About Replagal
FAQs
Replagal is a treatment for Fabry disease patients ages 7 and older. Shire (now owned by Takeda) developed it. Although ... Read more
March 2, 2021March 2, 2021
New Test Can Detect Antibodies That Limit ERT, Gene Therapy
News
Freeline Therapeutics has developed a more robust test to detect the presence of neutralizing antibodies that affect the effectiveness of ... Read more
February 26, 2021February 26, 2021
Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion
News, syndicated
Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years ... Read more
February 26, 2021February 26, 2021
Building Emotional Resilience When You Have Fabry Disease
Health Insights
Emotional resilience is the ability to adapt to stressful situations and cope with life’s ups and downs. Here’s how you ... Read more

April 9, 2021April 9, 2021

‘Break a Sweat for Fabry’ and Other Activities Mark Awareness Month

News

Activities ranging from sharing educational posters to “breaking a sweat” are underway to mark Fabry Disease Awareness Month, set aside each April to call attention to this rare genetic disorder. ... Read more

April 2, 2021April 2, 2021

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

News

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of ... Read more

March 26, 2021March 26, 2021

Fabrazyme’s Long-term Benefits to Kidneys, Other Organs Added to US Label

News

Long-term treatment with Fabrazyme (agalsidase beta), an enzyme replacement therapy for people with Fabry disease, slows the progression of kidney disease and significantly delays arriving at outcomes that include heart or kidney ... Read more

March 24, 2021March 23, 2021

Regaining My Strength and Stamina Following Heart Transplant

Columns, Fabry: A Road Less Traveled – a Column by Jerry Walter

I never imagined my first episode of irregular heartbeats, or arrhythmias, in the late 1970s would lead to getting a heart transplant decades later. When I had my first episode, ... Read more

March 19, 2021March 19, 2021

NanoGLA, Possibly More Effective ERT for Fabry, Named Orphan Drug in EU

News

NanoGLA — a potentially more effective version of enzyme replacement therapy that delivers the missing enzyme in Fabry disease through tiny fatty vesicles — has been designated an orphan drug by the European Commission. ... Read more

March 12, 2021

How Family Members Can Help Fabry Disease Patients

Health Insights

Fabry disease is a progressive, rare, genetic disorder that can lead to a number of health issues that affect a person’s everyday life. Following are some ways that family members ... Read more

March 12, 2021March 12, 2021

PRX-102 Safe and Effective Once Monthly Fabry Treatment, Phase 3 Data Show

News

A once-a-month regimen of the investigational enzyme replacement therapy (ERT) PRX-102 (pegunigalsidase alfa) is safe and effective at maintaining disease stability in adults with Fabry disease, top-line data from the ... Read more

March 8, 2021March 8, 2021

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

News, syndicated

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, ... Read more

March 5, 2021March 5, 2021

FAQs About Replagal

FAQs

Replagal is a treatment for Fabry disease patients ages 7 and older. Shire (now owned by Takeda) developed it. Although in use in many countries, the U.S. Food and Drug ... Read more

March 2, 2021March 2, 2021

New Test Can Detect Antibodies That Limit ERT, Gene Therapy

News

Freeline Therapeutics has developed a more robust test to detect the presence of neutralizing antibodies that affect the effectiveness of enzyme replacement therapy (ERT) or gene therapy in people with ... Read more